Around the Helix: Cell and Gene Therapy Company Updates – November 15, 2023

The cell and gene therapy sectors are growing exponentially, with new players emerging daily and much progress being made both in and out of the lab. CGTLive™’s Around the Helix is your chance to catch up with the latest news in cell and gene therapies, including partnerships, pipeline updates, and more.

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1. New Data Presented at AHA’s Scientific Sessions 2023

Catch up on CGTLive’s coverage of the American Heart Association’s (AHA) Scientific Sessions 2023, held November 10-13 in Philadelphia, Pennsylvania. Our coverage will continue throughout the week.

2. Adzynma Engineered ERT Therapy Approved for Prophylactic, On-Demand cTTP Treatment

The FDA has approved Takeda Pharmaceuticals’ Adzynma (TAK-755) as a prophylactic or on demand enzyme replacement therapy (ERT) in adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP).

3. Kyverna’s KYV-101 CAR T-Cell Therapy Expands Autoimmune Investigations Into Myasthenia Gravis

The FDA has granted investigational new drug (IND) clearance for an open-label, phase 2 trial of Kyverna Therapeutics’ KYV-101 chimeric antigen receptor T-cell (CAR-T) therapy in 10 patients with myasthenia gravis.

4. AviadoBio Gets FDA Clearance to Expand Trial of Frontotemporal Dementia Gene Therapy AVB-101

AviadoBio’s AVB-101, an investigational adeno-associated virus vector-based gene therapy intended to treat frontotemporal dementia associated with progranulin gene mutations, has received both clearance of its investigational new drug application and fast track designation from the FDA.

5. Jonas Wang, PhD, on the Advantages of Cord Blood-Derived Stem Cells in Regenerative Medicine

For World Cord Blood Day, recognized annually on November 15, the chairman and president of StemCyte discussed his thoughts on the importance of cord blood stem cells for the healthcare community.

6. Avid Bioservices Lends CDMO Support to CIRM-funded Partners

As part of a new partnership with the California Institute for Regenerative Medicine (CIRM), contract development and manufacturing organization (CDMO) Avid Bioservices will cover activities such as manufacturing and process and analytical development for CIRM-funded programs that are developing gene therapies.

7. Ohio State University Enlists CDMO Andelyn Biosciences

In a newly announced collaboration, Andelyn Biosciences will provide support in the form of manufacturing services, process optimization, and use of its platforms to the Ohio State University Gene Therapy Institute. The collaboration will focus on aiding the advancement of Ohio State University’s gene therapy programs for indications including Parkinson disease and amyotrophic lateral sclerosis.

8. Vittoria Biotherapeutics Garners $15 Million for CAR-T Program

The funding, which came in via private financing, will be put towards the ongoing development of VIPER-101, Vittoria’s autologous, dual population CD5-knockout CAR-T. "With a targeted focus on streamlined operations and an emphasis on cash efficiency, we plan to use this capital to advance our lead program, VIPER-101 - for the treatment of T-cell lymphoma - into the clinic in early 2024,” Nicholas Siciliano, PhD, the CEO of Vittoria, said in a statement.

9. Ajinomoto Co., Inc. To Bring Forge Biologics Into the Fold

Forge Biologics, which is both a CDMO focused on viral vectors and plasmids and a gene therapy developer with its own gene therapy product in clinical development for Krabbe disease, has agreed to be acquired by The Ajinomoto Group for $620 million in cash.

10. Arcellx and Kite Carve Out New Areas of Collaboration

Arcellx and Kite, a Gilead Company, which previously established a strategic partnership late last year, have agreed to expand their collaborative activities in several key areas. Notably, Kite has exercised an option related to Arcellx’s multiple myeloma (MM) T-cell therapy ACLX-001 and the companies have agreed to expand their collaboration on Arcellx's CART-ddBCMA, initially also focused on MM, into lymphoma indications.

11. Novartis Agrees to Shoulder Later-stage Development Activites for Legend Biotech’s DLL3-targeted CAR-T

Under a new agreement, Novartis will pay $100 million to Legend upfront, with milestone payments and tiered royalties potentially coming down the pike, for exclusive global rights related to certain DLL3-targeted CAR-T therapies Legend is developing. Most notable is LB2102, for which Legend has received FDA clearance to initiate a phase 1 clinical trial in extensive stage small cell lung cancer and large cell neuroendocrine carcinoma. As part of the deal, Novartis has the right to implement its T-Charge CAR-T cell therapy platform into the manufacturing of the CAR-T therapies covered under the agreement.

12. Landmark Bio Agrees to Manufacture CAR-T therapy for Galapagos’ Clinical Trial

Galapagos, which is developing CAR-T therapies for hematological cancers, has designed a decentralized manufacturing model intended to allow patients to receive autologous CAR T-cells at a median of 7 days following leukapheresis. Under the new agreement, Landmark will seek to implement this model during its manufacturing of clinical trial batches at its 44,000 square-foot development and manufacturing facility.